Claims for Patent: 11,058,668

Summary for Patent: 11,058,668

Title:	Methods of treating TTR amyloidosis using AG10
Abstract:	Described herein are methods for treating transthyretin (TTR) amyloidosis in a subject. The methods include specific dosing regimens that have great efficacy in treating the subjects and that are well tolerated in subjects.
Inventor(s):	Uma Sinha, Satish Rao
Assignee:	Eidos Therapeutics Inc
Application Number:	US16/361,587
Patent Claims:	1. A method of treating transthyretin (TTR) amyloidosis in a subject in need thereof, the method comprising orally administering to a subject in need thereof a total daily dosage of about 1,600 mg of Compound 1, having the formula: in HCl salt form, wherein Compound 1 is administered twice daily. 2. The method of claim 1, wherein said TTR amyloidosis is a disease or condition selected from the group consisting of familial amyloid polyneuropathy, familial amyloid cardiomyopathy, senile systemic amyloidosis, central amyloidosis, ocular amyloidosis, Leptomeningeal amyloidosis, oculoleptomeningeal amyloidosis, vitreous amyloidosis, gastrointestinal amyloidosis, neuropathic amyloidosis, non-neuropathic amyloidosis, non-hereditary amyloidosis, reactive/secondary amyloidosis, cerebral amyloidosis. 3. The method of claim 1, wherein said TTR amyloidosis is transthyretin amyloidosis (ATTR) cardiomyopathy. 4. The method of claim 1, wherein said TTR amyloidosis is transthyretin amyloidosis (ATTR) polyneuropathy. 5. The method of claim 3, wherein administration of a therapeutically effective amount of Compound 1 in a subject with ATTR cardiomyopathy improves the subject's performance in a six minute walk test as compared to subjects not receiving treatment. 6. The method of claim 3, wherein administration of a therapeutically effective amount of Compound 1 in a subject with ATTR cardiomyopathy decreases the frequency of cardiovascular-related hospitalizations as compared to subjects not receiving treatment. 7. The method of claim 3, wherein administration of a therapeutically effective amount of Compound 1 in a subject with ATTR cardiomyopathy decreases mortality as compared to subjects not receiving treatment. 8. The method of claim 4, wherein administration of a therapeutically effective amount of Compound 1 in a subject with ATTR polyneuropathy improves the Neuropathy Impairment Score (NIS) in said subject. 9. The method of claim 3, wherein said ATTR cardiomyopathy is wild-type ATTR cardiomyopathy (ATTRwt-CM). 10. The method of claim 3, wherein said ATTR cardiomyopathy is familial ATTR cardiomyopathy (ATTRm-CM). 11. The method of claim 4, wherein said ATTR polyneuropathy is wild-type ATTR polyneuropathy (ATTRwt-PN). 12. The method of claim 4, wherein said ATTR polyneuropathy is familial ATTR polyneuropathy (ATTRm-PN). 13. The method of claim 1, wherein administration of a therapeutically effective amount of Compound 1 increases transthyretin (TTR) blood serum concentration relative to a baseline level. 14. The method of claim 1, wherein administration of a therapeutically effective amount of Compound 1 increases transthyretin (TTR) blood serum concentration by at least 10% relative to a baseline level after 28 days of treatment.